Safety, Tolerability, and Efficacy of Viltolarsen in Boys with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial

Safety, Tolerability, and Efficacy of Viltolarsen in Boys with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial. …………… Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. JAMA Neurol.…

Effect of inclisiran, the siRNA against PCSK9, on platelets, immune cells and immunological biomarkers – a pre-specified analysis from ORION-1

Effect of inclisiran, the siRNA against PCSK9, on platelets, immune cells and immunological biomarkers – a pre-specified analysis from ORION-1 …………… Landmesser U, Haghikia A, Leiter LA, Wright RS, Kallend D, Wijngaard P, Stoekenbroek R, Kastelein JJP, Ray KK. Cardiovasc Res. 2020 Apr 3. PubMed

Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis.

Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis. …………… Sutherland JE, Hettinger JL, Chan A, Gilbert J, Warner GL, Davis WP. Nucleic Acid Ther. 2019 Dec 10. PubMed